BioMarin announced that valoctocogene roxaparvovec, for severe hemophilia A achieved pre-specified clinical criteria for regulatory review in the U.S. and Europe.

BioMarin Pharmaceutical announced that its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A achieved pre-specified clinical criteria for regulatory review in the U.S. and Europe. As of May 28, 2019, eight patients in the 20-patient cohort of the Phase III GENEr8-1 study achieved Factor VIII levels of 40 international units per deciliter (IU/dL), or more, at 23 to 26 weeks, meeting the pre-specified criteria for Factor VIII activity levels.

The company will meet with the FDA and European Medicines Agency (EMA) to review the phase III data and the other elements of a submission and intends to announce the timing for its planned marketing applications in 3Q 2019. As of the April 30, 2019 data cutoff, between weeks 23 to 26, in a cohort of the Phase III GENEr8-1 study of valoctocogene roxaparvovec dosed at 6e13 vg/kg, seven of 16 study participants reached or exceeded the pre-specified Factor VIII levels of 40 international units per deciliter using the chromogenic substrate (CS) assay. Subsequent to the April 30 cutoff, one additional participant met that criteria, bringing the total to eight participants.